Dystrophic Epidermolysis Bullosa Clinical Trial
Official title:
A Prospective, Longitudinal Assessment of Disease Severity in Subjects With Dystrophic Epidermolysis Bullosa (DEB)
Verified date | February 2013 |
Source | Lotus Tissue Repair, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Institutional Review Board |
Study type | Observational |
The objective of this study is to characterize the extent and severity of disease in subjects with DEB and the progression of disease over a timeframe relevant to interventional studies. The data from this study will be used to inform the study design and address statistical considerations of future treatment protocols.
Status | Withdrawn |
Enrollment | 0 |
Est. completion date | September 2014 |
Est. primary completion date | September 2014 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A and older |
Eligibility |
Eligibility Criteria - Subjects of any age (newborns included) may participate - Subjects over 18 years of age and parent(s)/legal guardian(s) of subjects <18 years of age must provide written informed consent prior to participating in the study and informed assent will be obtained from minors at least 7 years of age - Subjects must have a documented diagnosis of DEB based on clinical presentation and either skin biopsy results showing an absence or reduction in C7 or anchoring fibrils or genetic analysis showing a mutation in collagen, type VII, alpha 1 (Col7A1); alternatively, subjects must have a clinical diagnosis of DEB and a documented diagnosis of DEB (as above) in a first degree relative - No experimental systemic therapy for DEB including, but not limited to, bone marrow transplantation, systemic immune suppression, or experimental therapies that involve live cells which have the potential for systemic spread such as gene transfer, stem cell infusions or other cell type injections |
Observational Model: Cohort, Time Perspective: Prospective
Country | Name | City | State |
---|---|---|---|
United States | Stanford University School of Medicine | Palo Alto | California |
Lead Sponsor | Collaborator |
---|---|
Lotus Tissue Repair, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Characterize the progression of disease severity in subjects with DEB over 6 - 12 months. | Disease severity and its impact on quality of life and function will be investigated over a one year period at the following timepoints: upon enrollment, and at 1 to 2 weeks and 6 and 12 months after enrollment. | One year period | No |
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